Tenaya Therapeutics Reports First Quarter 2023 Financial Results and Provides Business Update
10 Maggio 2023 - 10:19PM
Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage
biotechnology company with a mission to discover, develop and
deliver potentially curative therapies that address the underlying
causes of heart disease, today reported financial results and
provided a corporate update for the first quarter ended March 31,
2023.
“Tenaya is making meaningful progress across multiple fronts of
our business while maintaining a solid cash position. We are
pleased to have recently received Fast Track designation for TN-201
and completed dosing in our Phase 1 trial of TN-301. We remain on
track to achieve our 2023 development and regulatory milestones for
TN-201, TN-301 and TN-401, including the release of clinical data
for TN-301 in the second half of 2023,” said Faraz Ali, Chief
Executive Officer of Tenaya. “In addition to our focus on achieving
near-term catalysts, we continue to invest in our future. We look
forward to highlighting the depth and breadth of our core
capabilities at the upcoming ASGCT conference. We have also added
leading industry and academic experts to our Scientific Advisory
Board and to our newly created Technical Advisory Board. These
efforts will collectively help Tenaya lay the foundation for
commercial-ready manufacturing for our existing product candidates
and support the expansion of Tenaya’s pipeline into new modalities,
including gene editing, so that we can bring the promise of genetic
medicines to individuals and families fighting heart disease.”
Business and Program Updates TN-201 – Gene
Therapy for MYBPC3-Associated Hypertrophic Cardiomyopathy
(HCM)Tenaya received notification that the U.S. Food and Drug
Administration (FDA) granted Fast Track designation to TN-201. The
FDA Fast Track program is designed to facilitate the development
and expedite the review of drug candidates intended to treat
serious conditions and for which nonclinical data demonstrates the
potential to address unmet medical need.
- In January 2023, the FDA notified Tenaya that clinical testing
of TN-201 may proceed based on review of the company’s
Investigational New Drug (IND) application.
- Tenaya plans to commence a Phase 1b multi-center, open-label,
dose-escalation clinical trial in the third quarter of 2023. The
Phase 1b clinical trial is designed to assess the safety,
tolerability and pharmacodynamics of a one-time intravenous
infusion of TN-201 in symptomatic adults with MYBPC3-associated
HCM. Initial data from the Phase 1b clinical trial are anticipated
in 2024.
TN-301 – Small Molecule HDAC6 Inhibitor for Heart Failure with
Preserved Ejection Fraction (HFpEF)
- Tenaya recently completed dosing of healthy participants in its
Phase 1 clinical trial. Initial target engagement, measured by the
biomarker tubulin acetylation, was achieved and no dose-limiting
toxicities were reported. The Phase 1 clinical trial was designed
to evaluate the safety, tolerability, pharmacokinetics and
pharmacodynamics of escalating oral doses of TN-301.
- Tenaya expects to report results from both the single and
multiple ascending dose stages of the Phase 1 clinical trial in the
second half of 2023.
TN-401 – Gene Therapy for PKP2-Associated Arrhythmogenic Right
Ventricular Cardiomyopathy (ARVC)
- Tenaya plans to submit an IND application for TN-401 to the FDA
in the second half of 2023. TN-401 is being developed for the
potential treatment of ARVC caused by PKP2 genetic mutations and is
designed to deliver a fully functional PKP2 gene to prevent
arrhythmia and reverse disease following a single dose.
Research Updates
- In May 2023, Tenaya researchers will present six abstracts at
the American Society of Gene and Cell Therapy (ASGCT) 26th Annual
Meeting detailing the company’s expanded capabilities for
manufacturing and discovering genetic medicines for heart disease.
Tenaya’s continued growth and optimization across its enabling
technologies supports current pipeline candidates and is expected
to increase opportunities for future programs. The presentations
will highlight innovations in manufacturing, capsid engineering and
gene editing.
Leadership
- Tenaya expanded its Scientific Advisory Board with the
appointments of Christine Edry Seidman, M.D., and Alexander Marson,
M.D., Ph.D.
- Dr. Seidman leads a laboratory that aims to define genetic
causes, mechanisms, treatments and cures for human heart disease.
Her work enabled development of the first mechanism-based treatment
for hypertrophic cardiomyopathy. Among her current roles, she
serves as the Director of the Cardiovascular Genetics Center at
Brigham and Women's Hospital, and a professor of genetics and
medicine at Harvard Medical School. Previously, Dr. Seidman
was a scientific co-founder of MyoKardia, a precision medicine
cardiovascular company focused on cardiomyopathies (acquired by
BMS).
- Dr. Marson’s research focuses on understanding the genetic
circuits that control human immune cell function and deploying
genome editing technologies such as CRISPR to create engineered
cells capable of detecting, resisting and eliminating disease. He
currently serves as Director of the Gladstone-UCSF Institute of
Genomic Immunology and professor of medicine at UCSF. He is also
co-founder of Spotlight Therapeutics, an in vivo genome editing
company using non-viral delivery.
- Tenaya announced the formation of a Technical Advisory Board
(TAB) to provide expert perspective on manufacturing, quality,
regulatory process development and analytical development
strategies across a range of modalities. Members of the TAB each
bring significant expertise in the manufacture of genetic medicines
and include:
- Mark Angelino, Ph.D., Venture Partner at Third Rock Ventures.
Dr. Angelino brings broad operational experience, most recently as
co-founder and Chief Operating Officer of Generation Bio, a
non-viral delivery genetics medicine company, and prior as the
Senior Vice President of Pharmaceutical Sciences at bluebird bio,
an ex vivo autologous lentiviral vector gene therapy company.
- Guangping Gao, Ph.D., Director of the Li Weibo Institute for
Rare Diseases and professor at UMass Chan Medical School. Dr. Gao
is widely recognized for his significant contributions to the field
of discovery and manufacturing of viral vector gene therapies for
rare diseases.
- Victoria Sluzky, Ph.D., is founder and principal of Lucid
Biotechnology Advisors with more than 30 years of biotechnology
industry experience. Dr. Sluzky formerly served as the Senior Vice
President of Technical Development at BioMarin Pharmaceutical where
she was directly involved in advancing product development in many
rare diseases, and providing expertise in quality, process and
assay development, formulation and regulatory strategy.
- Angela Thedinga, MBA, MPH, is presently a Board member of
Nkarta, Inc. Previously, Ms. Thedinga was the Chief Technology
Officer at Adverum and led manufacturing strategy and program
management at AveXis (now Novartis Gene Therapies).
First Quarter 2023 Financial Highlights
- Cash Position and updated cash guidance: As of
March 31, 2023, cash, cash equivalents and investments in
marketable securities were $173.6 million. Tenaya expects current
cash, cash equivalents and investments in marketable securities
(current and noncurrent) will be sufficient to fund the company
into the first half of 2025.
- Research & Development (R&D) Expenses:
R&D expenses for the quarter ended March 31, 2023, were $25.6
million. Non-cash stock-based compensation included in R&D
expense was $1.6 million for the quarter ended March 31, 2023.
- General & Administrative (G&A)
Expenses: G&A expenses for the quarter ended March 31,
2023, were $8.1 million. Non-cash stock-based compensation included
in G&A expense was $1.9 million for the quarter ended March 31,
2023.
- Net Loss: Net loss for the quarter ended March
31, 2023, was $31.7 million, or $0.43 per share.
About Tenaya Therapeutics Tenaya Therapeutics
is a clinical-stage biotechnology company committed to a bold
mission: to discover, develop and deliver potentially curative
therapies that address the underlying drivers of heart disease.
Leveraging its integrated and interrelated Gene Therapy, Cellular
Regeneration and Precision Medicine platforms and proprietary core
capabilities, the company is advancing a pipeline of novel
therapies with diverse treatment modalities for rare genetic
cardiovascular disorders and more prevalent heart conditions.
Tenaya’s most advanced candidates include TN-201, a gene therapy
for MYBPC3-associated hypertrophic cardiomyopathy (HCM), TN-401, a
gene therapy for PKP2-associated arrhythmogenic right ventricular
cardiomyopathy (ARVC), and TN-301, a small molecule HDAC6 inhibitor
being initially developed for heart failure with preserved ejection
fraction (HFpEF). Tenaya also has multiple early-stage programs
progressing through preclinical development. For more information,
visit www.tenayatherapeutics.com.
Forward-looking Statement. This press release
contains forward-looking statements as that term is defined in
Section 27A of the Securities Act of 1933 and Section 21E of the
Securities Exchange Act of 1934. Statements in this press release
that are not purely historical are forward-looking statements.
Words such as “expected,” “look forward,” “plans,” “anticipated,”
“expects,” “will,” and similar expressions are intended to identify
forward-looking statements. Such forward-looking statements
include, among other things, statements regarding 2023 development
and regulatory milestones; Tenaya’s plan to present abstracts
covering manufacturing, capsid engineering and gene editing at the
upcoming ASGCT conference; expected timing for, commencement of
dosing in the Phase 1b clinical trial evaluating TN-201 and
availability of initial data from the trial, data from the SAD and
MAD stages of the Phase 1 clinical trial evaluating TN-301 and the
submission of the TN-401 IND; the potential for Tenaya’s future
programs; the sufficiency of Tenaya’s cash resources to fund the
company into the first half 2025; and statements by Tenaya’s chief
executive officer. The forward-looking statements contained herein
are based upon Tenaya’s current expectations and involve
assumptions that may never materialize or may prove to be
incorrect. These forward-looking statements are neither promises
nor guarantees and are subject to a variety of risks and
uncertainties, including but not limited to: Tenaya’s ability to
develop, initiate or complete preclinical studies and clinical
trials for its product candidates; the timing, scope and likelihood
of regulatory filings and approvals; the availability of data at
the referenced times; risks associated with the process of
discovering, developing and commercializing drugs that are safe and
effective for use as human therapeutics and operating as an early
stage company; Tenaya’s ability to raise any additional funding it
will need to continue to pursue its business and product
development plans; the potential for any clinical trial results to
differ from preclinical, interim, preliminary, topline or expected
results; Tenaya’s ability to successfully operate a manufacturing
facility for clinical or commercial supply; Tenaya’s
commercialization and marketing capabilities and strategy; the loss
of key scientific or management personnel; competition in the
industry in which Tenaya operates; Tenaya’s reliance on third
parties; Tenaya’s ability to obtain and maintain intellectual
property protection for its product candidates; general economic
and market conditions; and other risks. Information regarding the
foregoing and additional risks may be found in the section entitled
“Risk Factors” in documents that Tenaya files from time to time
with the Securities and Exchange Commission. These forward-looking
statements are made as of the date of this press release, and
Tenaya assumes no obligation to update or revise any
forward-looking statements, whether as a result of new information,
future events or otherwise, except as required by law.
Contacts
Investors |
Media |
Michelle CorralVice
President, Investor Relationship andCorporate Communications Tenaya
Therapeutics IR@tenayathera.com |
Wendy Ryan Ten Bridge
Communications Wendy@tenbridgecommunications.com |
TENAYA THERAPEUTICS, INC. Condensed
Statements of Operations (In thousands, except
share and per share data)
(Unaudited) |
|
|
|
Three Months Ended March 31, |
|
|
|
2023 |
|
|
2022 |
|
Operating
expenses: |
|
|
|
|
|
|
Research and
development |
|
$ |
25,605 |
|
|
$ |
24,155 |
|
General and
administrative |
|
|
8,118 |
|
|
|
6,999 |
|
Total operating expenses |
|
|
33,723 |
|
|
|
31,154 |
|
Loss from
operations |
|
|
(33,723 |
) |
|
|
(31,154 |
) |
Other income
(expense), net: |
|
|
|
|
|
|
Interest income |
|
|
1,973 |
|
|
|
99 |
|
Other income
(expense), net |
|
|
13 |
|
|
|
(1 |
) |
Total other income (expense), net |
|
|
1,986 |
|
|
|
98 |
|
Net loss before income
tax expense |
|
|
(31,737 |
) |
|
|
(31,056 |
) |
Income tax
expense |
|
|
— |
|
|
|
— |
|
Net loss |
|
$ |
(31,737 |
) |
|
$ |
(31,056 |
) |
Net loss per share,
basic and diluted |
|
$ |
(0.43 |
) |
|
$ |
(0.75 |
) |
Weighted-average
shares used in computing net loss per share, basic and diluted |
|
|
73,097,889 |
|
|
|
41,267,990 |
|
TENAYA THERAPEUTICS, INC.Condensed Balance
Sheet Data (In
thousands)(Unaudited) |
|
|
|
|
|
|
|
|
|
March 31, |
|
|
December 31, |
|
|
|
2023 |
|
|
2022 |
|
Cash, cash equivalents
and marketable securities |
|
$ |
173,635 |
|
|
$ |
204,230 |
|
Total assets |
|
$ |
245,341 |
|
|
$ |
278,945 |
|
Total liabilities |
|
$ |
29,929 |
|
|
$ |
35,569 |
|
Total liabilities and
stockholders’ equity |
|
$ |
245,341 |
|
|
$ |
278,945 |
|
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